CA2901676C - Methodes et compositions pour ameliorer une disruption genique a mediation nuclease - Google Patents

Methodes et compositions pour ameliorer une disruption genique a mediation nuclease Download PDF

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Publication number
CA2901676C
CA2901676C CA2901676A CA2901676A CA2901676C CA 2901676 C CA2901676 C CA 2901676C CA 2901676 A CA2901676 A CA 2901676A CA 2901676 A CA2901676 A CA 2901676A CA 2901676 C CA2901676 C CA 2901676C
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dna
cell
nuclease
cells
protein
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CA2901676A1 (fr
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Fuqiang Chen
Qiaohua KANG
Thomas Wechsler
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Sigma Aldrich Co LLC
Sangamo Therapeutics Inc
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Sigma Aldrich Co LLC
Sangamo Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/502—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with carbocyclic ring systems, e.g. cinnoline, phthalazine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

L'invention concerne des méthodes et des compositions pour accroître une modification génomique à médiation nucléase au moyen d'inhibiteurs de réparation d'ADN.
CA2901676A 2013-02-25 2014-02-24 Methodes et compositions pour ameliorer une disruption genique a mediation nuclease Active CA2901676C (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361769038P 2013-02-25 2013-02-25
US61/769,038 2013-02-25
PCT/US2014/018047 WO2014130955A1 (fr) 2013-02-25 2014-02-24 Méthodes et compositions pour améliorer une disruption génique à médiation nucléase

Publications (2)

Publication Number Publication Date
CA2901676A1 CA2901676A1 (fr) 2014-08-28
CA2901676C true CA2901676C (fr) 2023-08-22

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Family Applications (1)

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CA2901676A Active CA2901676C (fr) 2013-02-25 2014-02-24 Methodes et compositions pour ameliorer une disruption genique a mediation nuclease

Country Status (6)

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US (1) US10227610B2 (fr)
EP (1) EP2958996B1 (fr)
JP (2) JP6491113B2 (fr)
AU (1) AU2014218621B2 (fr)
CA (1) CA2901676C (fr)
WO (1) WO2014130955A1 (fr)

Families Citing this family (145)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2831144A1 (fr) 2011-03-23 2012-09-27 Pioneer Hi-Bred International, Inc. Procedes de production d'un locus complexe de caracteristique transgenique
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
SG11201406547YA (en) 2012-04-25 2014-11-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
WO2014143381A1 (fr) * 2013-03-09 2014-09-18 Agilent Technologies, Inc. Procédé de modification in vivo de grande séquences utilisant plusieurs sélections crispr/cas d'événements de recombinaison
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
WO2014204578A1 (fr) 2013-06-21 2014-12-24 The General Hospital Corporation Utilisation de nucléases foki à guidage arn (rfn) afin d'augmenter la spécificité pour l'édition de génome par guidage arn
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
CN120574876A (zh) 2013-08-22 2025-09-02 纳幕尔杜邦公司 使用向导rna/cas内切核酸酶系统的植物基因组修饰及其使用方法
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
EP3988649B1 (fr) 2013-09-18 2024-11-27 Kymab Limited Procédés, cellules et organismes
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CN106604994B (zh) 2014-06-23 2021-12-14 通用医疗公司 通过测序评估的DSBs的全基因组无偏鉴定(GUIDE-Seq)
US10676754B2 (en) 2014-07-11 2020-06-09 E I Du Pont De Nemours And Company Compositions and methods for producing plants resistant to glyphosate herbicide
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
MX2017002930A (es) 2014-09-12 2017-06-06 Du Pont Generacion de sitios de integracion especifica de sitio para loci de rasgos complejos en maiz y soja, y metodos de uso.
EP3201340B1 (fr) 2014-10-01 2020-12-02 The General Hospital Corporation Procédés destinés à augmenter l'efficacité de la réparation par homologie induite par nucléase
EP4464338A3 (fr) * 2014-11-07 2025-02-12 Editas Medicine, Inc. Systèmes pour améliorer l'édition génomique médiée par crispr/cas
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
CN116059378A (zh) 2014-12-10 2023-05-05 明尼苏达大学董事会 用于治疗疾病的遗传修饰的细胞、组织和器官
EP3245232B1 (fr) 2015-01-12 2021-04-21 The Regents of The University of California Cas9 hétérodimère et procédés d'utilisation associés
EP3250689B1 (fr) 2015-01-28 2020-11-04 The Regents of The University of California Procédés et compositions pour le marquage d'un acide nucléique cible monocaténaire
KR20230156800A (ko) 2015-03-03 2023-11-14 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
BR112017017260A2 (pt) 2015-03-27 2018-04-17 Du Pont construções de dna, vetor, célula, plantas, semente, método de expressão de rna e método de modificação de local alvo
AU2016263026A1 (en) 2015-05-15 2017-11-09 Pioneer Hi-Bred International, Inc. Guide RNA/Cas endonuclease systems
EP3095870A1 (fr) * 2015-05-19 2016-11-23 Kws Saat Se Procédés pour la transformation in planta de plantes et procédés de fabrication basés sur ceux-ci et produits pouvant être obtenus à partir de ceux-ci
WO2016196655A1 (fr) 2015-06-03 2016-12-08 The Regents Of The University Of California Variants de cas9 et procédés d'utilisation associés
EP3303585A4 (fr) 2015-06-03 2018-10-31 Board of Regents of the University of Nebraska Traitement des données d'une séquence d'adn utilisant un adn monocaténaire
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
JP6799586B2 (ja) 2015-08-28 2020-12-16 ザ ジェネラル ホスピタル コーポレイション 遺伝子操作ＣＲＩＳＰＲ−Ｃａｓ９ヌクレアーゼ
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
WO2017040709A1 (fr) * 2015-08-31 2017-03-09 Caribou Biosciences, Inc. Réparation d'acide nucléique dirigée
WO2017044843A1 (fr) 2015-09-11 2017-03-16 The General Hospital Corporation Interrogation complète de dsb nucléasiques et séquençage (find-seq)
JP2018529353A (ja) 2015-09-30 2018-10-11 ザ ジェネラル ホスピタル コーポレイション 配列決定法による切断事象の包括的生体外報告（ＣＩＲＣＬＥ−ｓｅｑ）
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
DK3387134T3 (da) 2015-12-11 2020-12-21 Danisco Us Inc Fremgangsmåder og sammensætninger til øget nukleasemedieret genommodifikation og reducerede virkninger uden for målstedet
EP3390631B1 (fr) 2015-12-18 2020-04-08 Danisco US Inc. Procédés et compositions pour l'expression d'arn de guidage à base d'arnt
US20190249172A1 (en) 2016-02-18 2019-08-15 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
CN109414414A (zh) 2016-03-16 2019-03-01 戴维·格拉德斯通研究所 用于治疗肥胖症和/或糖尿病以及用于鉴定候选治疗剂的方法和组合物
US11597924B2 (en) * 2016-03-25 2023-03-07 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
JP6925003B2 (ja) * 2016-05-10 2021-08-25 公立大学法人横浜市立大学 非相同末端連結欠損細胞及びその利用
WO2017205503A1 (fr) 2016-05-24 2017-11-30 Indiana University Research And Technology Corporation Inhibiteurs de ku et leur utilisation
EP3464594A1 (fr) 2016-06-01 2019-04-10 Kws Saat Se Séquences d'acides nucléiques hybrides destinées à l'ingénierie génomique
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (fr) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Protéines de fusion cas9-recombinase programmables et utilisations associées
AU2017312132A1 (en) * 2016-08-19 2019-03-21 Bluebird Bio, Inc. Genome editing enhancers
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US20200199599A1 (en) 2016-09-23 2020-06-25 Dsm Ip Assets B.V. A guide-rna expression system for a host cell
MX2019003674A (es) 2016-09-30 2021-01-08 Univ California Enzimas modificadoras de ácido nucleico guiadas por arn y métodos de uso de estas.
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
CN110520530A (zh) 2016-10-18 2019-11-29 明尼苏达大学董事会 肿瘤浸润性淋巴细胞和治疗方法
AU2017378427A1 (en) 2016-12-14 2019-06-20 Ligandal, Inc. Methods and compositions for nucleic acid and protein payload delivery
WO2018119359A1 (fr) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Édition du gène récepteur ccr5 pour protéger contre l'infection par le vih
CA3052099A1 (fr) 2017-01-30 2018-08-02 Mathias LABS Liaison de modele de reparation a des endonucleases pour ingenierie genomique
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3592853A1 (fr) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression de la douleur par édition de gène
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
CN110462044A (zh) 2017-04-06 2019-11-15 帝斯曼知识产权资产管理有限公司 自引导整合构建体(sgic)
KR102537447B1 (ko) * 2017-04-10 2023-05-30 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. 유전체 편집 효율 증가를 위한 화합물
EP3388517A1 (fr) * 2017-04-10 2018-10-17 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Composés pour accroître l'efficacité d'édition du génome
US11591589B2 (en) 2017-04-21 2023-02-28 The General Hospital Corporation Variants of Cpf1 (Cas12a) with altered PAM specificity
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3630849A4 (fr) 2017-05-25 2021-01-13 The General Hospital Corporation Architectures d'éditeur de base bipartite (bbe) et édition de doigt de zinc de type ii-c-cas9
EP3645021A4 (fr) 2017-06-30 2021-04-21 Intima Bioscience, Inc. Vecteurs viraux adéno-associés destinés à la thérapie génique
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3663310A4 (fr) 2017-08-04 2021-08-11 Peking University Rvd de tale reconnaissant spécifiquement une base d'adn modifiée par méthylation et application correspondante
WO2019028686A1 (fr) 2017-08-08 2019-02-14 北京大学 Procédé d'inactivation génique
CA3073448A1 (fr) 2017-08-23 2019-02-28 The General Hospital Corporation Nucleases crispr-cas9 genetiquement modifiees presentant une specificite pam modifiee
EP3676376B1 (fr) 2017-08-30 2025-01-15 President and Fellows of Harvard College Éditeurs de bases à haut rendement comprenant une gam
EP3679144A1 (fr) * 2017-09-08 2020-07-15 Life Technologies Corporation Méthodes de recombinaison homologue améliorée et compositions associées
EP3679143A1 (fr) 2017-09-08 2020-07-15 Life Technologies Corporation Procédés de recombinaison homologue améliorés et compositions associées
WO2019075197A1 (fr) 2017-10-11 2019-04-18 The General Hospital Corporation Procédés de détection de désamination génomique parasite et spécifique de site induite par des technologies d'édition de base
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
EP3704255A1 (fr) 2017-10-30 2020-09-09 KWS SAAT SE & Co. KGaA Nouvelles stratégies d'édition génomique de précision
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019012162A2 (fr) 2017-12-20 2019-01-17 Dsm Ip Assets B.V. Méthode d'édition génomique dans une cellule hôte
JP7466448B2 (ja) * 2018-01-17 2024-04-12 バーテックス ファーマシューティカルズ インコーポレイテッド Ｄｎａ－ｐｋ阻害剤
BR112020013626A2 (pt) 2018-01-17 2020-12-01 Vertex Pharmaceuticals Incorporated compostos de quinoxalinona, composições, métodos e kits para aumentar a eficiência de edição de genoma
MA51619A (fr) 2018-01-17 2021-04-14 Vertex Pharma Inhibiteurs de la protéine kinase dépendante de l'adn
WO2019165168A1 (fr) 2018-02-23 2019-08-29 Pioneer Hi-Bred International, Inc. Nouveaux orthologues de cas9
US11898203B2 (en) 2018-04-17 2024-02-13 The General Hospital Corporation Highly sensitive in vitro assays to define substrate preferences and sites of nucleic-acid binding, modifying, and cleaving agents
CN112088215A (zh) 2018-05-09 2020-12-15 帝斯曼知识产权资产管理有限公司 Crispr瞬时表达构建体(ctec)
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
WO2019224603A1 (fr) * 2018-05-24 2019-11-28 Crispr Therapeutics Ag Procédés et compositions pour la délétion de gène efficace
CN110592141B (zh) 2018-06-13 2023-07-07 中国科学院上海有机化学研究所 用于调控基因编辑效率的化合物及其应用
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
JP2021530212A (ja) 2018-07-13 2021-11-11 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California レトロトランスポゾンベースの送達媒体及びその使用方法
US12275964B2 (en) 2018-08-22 2025-04-15 The Regents Of The University Of California Variant type V CRISPR/Cas effector polypeptides and methods of use thereof
AU2019360270B2 (en) 2018-10-18 2025-08-07 Intellia Therapeutics, Inc. Compositions and methods for expressing factor IX.
US11407995B1 (en) 2018-10-26 2022-08-09 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
WO2020092453A1 (fr) 2018-10-29 2020-05-07 The Broad Institute, Inc. Éditeurs de nucléobases comprenant geocas9 et utilisations associées
US11434477B1 (en) 2018-11-02 2022-09-06 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
WO2020097445A1 (fr) 2018-11-09 2020-05-14 Inari Agriculture, Inc. Nucléases guidées par arn et protéines de liaison à l'adn
US20220056460A1 (en) 2018-12-05 2022-02-24 Dsm Ip Assets B.V. Crispr guide-rna expression strategies for multiplex genome engineering
WO2020123887A2 (fr) 2018-12-14 2020-06-18 Pioneer Hi-Bred International, Inc. Nouveaux systèmes crispr-cas d'édition du génome
US20220154220A1 (en) 2018-12-21 2022-05-19 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Small molecules for increasing precise genome editing efficiency
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
WO2020163856A1 (fr) 2019-02-10 2020-08-13 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Mitochondrie modifiée et ses méthodes d'utilisation
MX2021010559A (es) 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
WO2020191233A1 (fr) 2019-03-19 2020-09-24 The Broad Institute, Inc. Procédés et compositions pour l'édition de séquences nucléotidiques
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020224987A1 (fr) 2019-05-06 2020-11-12 Dsm Ip Assets B.V. Donneur de crispr à plusieurs parties
EP4022057A1 (fr) * 2019-08-27 2022-07-06 Vertex Pharmaceuticals Incorporated Compositions et procédés pour le traitement de troubles associés à l'adn répétitif
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4055170A1 (fr) 2019-11-04 2022-09-14 DSM IP Assets B.V. Transfection à faible volume
BR112022011372A2 (pt) 2019-12-10 2022-08-23 Univ Indiana Trustees Inibidores de interação de proteína a de replicação (rpa)-dna
JP7846007B2 (ja) 2019-12-18 2026-04-14 ノバルティス アーゲー 異常ヘモグロビン症の治療用組成物及び方法
WO2021151085A2 (fr) 2020-01-24 2021-07-29 The General Hospital Corporation Enzymes crispr-cas ayant une activité sur cible améliorée
WO2021151073A2 (fr) 2020-01-24 2021-07-29 The General Hospital Corporation Ciblage de génome non contraint avec des variants de crispr-cas9 génétiquement modifiés presque sans pam
JP7649562B2 (ja) * 2020-02-19 2025-03-21 国立大学法人京都大学 ゲノム編集された細胞の製造方法及びそのためのキット
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
US20230183751A1 (en) * 2020-05-21 2023-06-15 Oxford Genetics Limited Hdr enhancers
CN111662925A (zh) * 2020-06-29 2020-09-15 中国农业科学院作物科学研究所 尼克酰胺在提高小麦基因编辑效率中的应用
TW202302848A (zh) * 2021-02-26 2023-01-16 美商維泰克斯製藥公司 以crispr/sacas9治療第1型肌強直性營養不良之組合物及方法
EP4298221A1 (fr) * 2021-02-26 2024-01-03 Vertex Pharmaceuticals Incorporated Compositions et méthodes pour le traitement de la dystrophie myotonique de type 1 avec crispr/slucas9
US20250127809A1 (en) 2021-06-23 2025-04-24 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
EP4596696A3 (fr) 2021-09-28 2025-08-27 Acrigen Biosciences Compositions et procédés pour modifications d'acide nucléique
CA3237482A1 (fr) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Edition precise du genome a l'aide de retrons
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
WO2023141602A2 (fr) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Rétrons modifiés et méthodes d'utilisation
CN114717263A (zh) * 2022-04-29 2022-07-08 河北科技大学 高同源重组率的细胞系的制备方法
CN115227834B (zh) * 2022-06-02 2025-11-11 中国人民解放军海军军医大学 通过基因编辑技术联合dna损伤修复抑制剂特异杀伤癌细胞的方法
JP2025525569A (ja) 2022-07-18 2025-08-05 レナゲード セラピューティクス マネージメント インコーポレイテッド 遺伝子編集成分、システム、及び使用方法
WO2024044723A1 (fr) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Rétrons modifiés et méthodes d'utilisation
KR20250027489A (ko) * 2023-08-16 2025-02-26 기초과학연구원 단일가닥 절단 활성을 가지는 CRISPR/Cas 시스템과 PARP 억제제를 이용하는 세포 사멸 방법
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
AU2024357861A1 (en) 2023-10-12 2026-04-23 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025155753A2 (fr) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Système, guides et procédés d'édition de gènes améliorés
WO2025174765A1 (fr) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Nanoparticules lipidiques comprenant des molécules d'arn codant destinées à être utilisées dans l'édition génique et comme vaccins et agents thérapeutiques
WO2026006542A2 (fr) 2024-06-26 2026-01-02 Yale University Compositions et méthodes de réactivation basée sur crispr/cas9 du syndrome d'angelman humain
WO2026005012A1 (fr) * 2024-06-28 2026-01-02 学校法人自治医科大学 Procédé d'amélioration de l'efficacité de recombinaison homologue dans l'édition génomique in vitro et in vivo

Family Cites Families (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE398679T1 (de) 1992-07-07 2008-07-15 Japan Tobacco Inc Verfahren zur transformation einer monokotyledon pflanze
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5928638A (en) 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
AR014491A1 (es) 1998-01-29 2001-02-28 Dow Agrosciences Llc Metodo para obtener plantas transgenicas fertiles de gossypium hirsutum.
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
AU776576B2 (en) 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US6689558B2 (en) 2000-02-08 2004-02-10 Sangamo Biosciences, Inc. Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
CA2410516A1 (fr) 2000-05-31 2001-12-06 Chiron Corporation Compositions et procedes servant a traiter des maladies neoplasiques au moyen d'agents de sensibilisation a la chimiotherapie et aux rayonnements
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Ｃｎｎについての亜鉛フィンガー結合ドメイン
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
WO2003087341A2 (fr) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Mutagenese chromosomique ciblee au moyen de nucleases en doigt a zinc
WO2003080809A2 (fr) 2002-03-21 2003-10-02 Sangamo Biosciences, Inc. Procedes et compositions permettant d'utiliser des endonucleases a doigts de zinc pour ameliorer la recombinaison homologue
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP2806025B1 (fr) 2002-09-05 2019-04-03 California Institute of Technology Utilisation de nucléases à doigt de zinc pour stimuler le ciblage de gènes
US20070134796A1 (en) 2005-07-26 2007-06-14 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
PL2305221T3 (pl) * 2003-12-01 2015-11-30 Kudos Pharm Ltd Inhibitory naprawy uszkodzeń DNA w leczeniu raka
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7253273B2 (en) 2004-04-08 2007-08-07 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
AU2005232665B2 (en) 2004-04-08 2010-05-13 Sangamo Therapeutics, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
US20070155014A1 (en) 2005-07-20 2007-07-05 Invitrogen Corporation Methods for increasing efficiency of homologous recombination
ES2582091T3 (es) * 2005-10-18 2016-09-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
EP2027262B1 (fr) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Demi-domaines résultant de clivage par génie génétique
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
CN104072561B (zh) 2007-06-19 2017-12-22 路易斯安那州州立大学及农业机械学院管理委员会 信使rna帽的抗‑反向硫代磷酸类似物的合成和用途
EP2171052B1 (fr) 2007-07-12 2014-08-27 Sangamo BioSciences, Inc. Procédés et compositions pour inactiver l'expression génique d'alpha-1,6-fucosyltransférase (fut 8)
HRP20161004T1 (hr) 2007-09-27 2016-10-21 Dow Agrosciences Llc Tehnološki konstruirani proteini cinkovi prsti koji imaju kao cilj gene za sintazu 5-enolpiruvilshikimat-3-fosfata
AU2008305590A1 (en) 2007-09-27 2009-04-02 Sangamo Therapeutics, Inc. Genomic editing in zebrafish using zinc finger nucleases
EP2205752B1 (fr) 2007-10-25 2016-08-10 Sangamo BioSciences, Inc. Procédés et compositions pour une intégration ciblée
CA2720903C (fr) 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Constructions donneuses lineaires pour une integration ciblee
WO2009151591A2 (fr) 2008-06-10 2009-12-17 Sangamo Biosciences, Inc. Procédés et compositions pour la génération de lignées cellulaires déficientes en bax et bak
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
JP5756016B2 (ja) 2008-10-29 2015-07-29 サンガモ バイオサイエンシーズ， インコーポレイテッド グルタミンシンセターゼ遺伝子の発現を不活性化する方法及び組成物
EP2352369B1 (fr) 2008-12-04 2017-04-26 Sangamo BioSciences, Inc. Édition de génome chez des rats au moyen de nucléases en doigt de zinc
EP2206723A1 (fr) 2009-01-12 2010-07-14 Bonas, Ulla Domaines modulaires de liaison à l'ADN
EP2408921B1 (fr) 2009-03-20 2017-04-19 Sangamo BioSciences, Inc. Modification de cxcr4 en utilisant des protéines à doigt de zinc modifiées
JP5940977B2 (ja) 2009-08-11 2016-06-29 サンガモ バイオサイエンシーズ， インコーポレイテッド 標的改変によるホモ接合生物
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
WO2011072246A2 (fr) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Modification de l'adn induite par l'effecteur tal
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
EP2660318A1 (fr) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Modification génomique ciblée de molécules partiellement donneuses à simple brin
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
US9044492B2 (en) * 2011-02-04 2015-06-02 Cellectis Sa Method for modulating the efficiency of double-strand break-induced mutagenesis
WO2012168307A2 (fr) 2011-06-07 2012-12-13 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Efficacité améliorée de recombinaison par inhibition d'une réparation de l'adn par nhej
CA2848417C (fr) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Procedes et compositions de regulation de l'expression d'un transgene
CA3099582A1 (fr) 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Procedes et compositions pour la modification du locus hprt
EP3196301B1 (fr) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Compositions et procédés pour le traitement de maladies génétiques monofactorielles
SG10201701601WA (en) 2012-08-29 2017-04-27 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
WO2014127287A1 (fr) * 2013-02-14 2014-08-21 Massachusetts Institute Of Technology Procédé pour la mutagenèse ciblée in vivo
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物

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